An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias

The purpose of this study is to learn how Intravenous Immune Globulin (IVIG) will affect Spinocerebellar Ataxia (SCA) symptoms and how it will affect motor and nervous system function in participants Subtypes of SCA to be examined will include SCA types 1, 2, 3, 6, 10 and 11.

Study Overview

• Status: Unknown
• Conditions: Spinocerebellar Ataxias
• Intervention / Treatment: Drug: Intravenous Immune Globulin (IVIG)

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Mary Freeman, LPN; Phone Number: 813-974-5909

Study Locations

• United States
  • Florida
    • Tampa, Florida, United States, 33612
      • Recruiting
      • University of South Florida
      • Contact: Mary Freeman, LPN; Phone Number: 813-974-5909
      • Contact: Tanya Aranca, BS; Phone Number: 813-974-5909

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Outpatients with SCA types 1, 2, 3, 6, 10, or 11, diagnosed by a movement disorder specialist.
• Age 18 years to 80 years.
• Able to ambulate with or without assistance for 30 feet.
• Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
• Serum creatine kinase, complete metabolic panel, complete blood count, liver function tests, renal function tests, platelets and EKG do not reveal clinically significant abnormalities (results obtained from primary care physician and dated within the past 6 months or obtained at screening visit).
• Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
• Diagnosis of peripheral neuropathy. See exclusion criteria 3 for specific types of peripheral neuropathy to be excluded.
• Throughout the study, all possible efforts will be made to maintain subject levels of activity, exercise or physical therapy.
• Subject permission (informed consent).

Exclusion Criteria:

• Any unstable illness that in the investigator's opinion precludes participation in this study.
• Use of any investigational product within the past 30 days.
• Presence of diabetes (as determined by blood glucose labs within the past 6 months), nutritional deficiency causing neuropathy (vitamin B1, 3, 6, and 12 or vitamin E), injuries, autoimmune disorders (HIV, lupus, pediatric Guillain-Barre syndrome, neurosarcoidosis, monoclonal gammopathy), tumors, infections (leprosy), exposures to toxins (alcohol, arsenic, mercury), thyroid disease or hereditary causes (cerebral amyloid angiopathy) known to result in the presence of peripheral neuropathy.
• Dementia or other psychiatric illness that prevents the subject from giving informed consent (MMSE less than 25).
• Legal incapacity or limited legal capacity.
• Presence of severe renal disease (estimated creatinine clearance <50 mL/min) or hepatic disease (as evidenced by labs reported within the past 6 months).
• Clinically significantly abnormal white blood cell count, hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
• Immunoglobulin A, Vitamin B (1, 3, 6, or 12), vitamin E or folate deficiencies (evidenced by screening lab evaluations).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Intravenous Immune Globulin (IVIG)

Drug: Intravenous Immune Globulin (IVIG); IVIG will be infused over a course of five days in the form of GAMMAGARD LIQUID 10% solution, available from Baxter. For neurological and autoimmune diseases 2 grams per kilogram of body weight is implemented for three months over a five day course once a month. There is very limited reliable dose ranging data for IVIG in the treatment of any condition, and most dosing has been empiric. In our experience, we have empirically observed a more potent immunomodulatory effect from "induction dose" IVIG (2 g/kg) continued each month, than with the "booster dose" maintenance dose of 1gm/kg. Though there is no category one evidence to support this practice, neither is there such evidence to refute it. Additionally, results from previous trials of IVIG in SCAs show this dosage to be relatively safe and effective at this rate of infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in Scale for the Assessment and Rating Ataxia (SARA)	The primary outcomes will be the changes in the patient's SARA total score and frequency and severity of adverse events.	Will be assesed at abseline, day 14, day28 and day 56.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
clinician and patient global impression of improvement (CGI and PGI)	Secondary outcome measures will include changes in the following scales between baseline and study endpoint: clinician and patient global impression of improvement (CGI and PGI); neurologic dysfunction as assessed by STAND scores; 9-hole peg test times.	Will be assesed at abseline, day 14, day28 and day 56.
Neurologic dysfunction as assessed by STAND scores	Secondary outcome measures will include changes in the following scales between baseline and study endpoint: clinician and patient global impression of improvement (CGI and PGI); neurologic dysfunction as assessed by STAND scores; 9-hole peg test times.	Will be assesed at abseline, day 14, day28 and day 56.
9-hole peg test	Secondary outcome measures will include changes in the following scales between baseline and study endpoint: clinician and patient global impression of improvement (CGI and PGI); neurologic dysfunction as assessed by STAND scores; 9-hole peg test times.	Will be assesed at abseline, day 14, day28 and day 56.

Collaborators and Investigators

• Sponsor: University of South Florida
• Collaborators: Baxter Healthcare Corporation

Study record dates

Study Major Dates

• Study Start: April 1, 2015
• Primary Completion (Anticipated): December 1, 2016

Study Registration Dates

• First Submitted: November 5, 2014
• First Submitted That Met QC Criteria: November 7, 2014
• First Posted (Estimate): November 10, 2014

Study Record Updates

• Last Update Posted (Estimate): June 8, 2016
• Last Update Submitted That Met QC Criteria: June 6, 2016
• Last Verified: June 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations; Physiological Effects of Drugs; Immunologic Factors; Antibodies; Immunoglobulins; Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin
• Other Study ID Numbers: SCA IVIG 2014